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Clinically relevant end points and new drug approvals for myeloma

Leukemia volume 22, pages 231–239 (2008)Cite this article

Abstract

This manuscript summarizes the recommendations of the American Society of Hematology/US Food and Drug Administration Workshop on Clinical Endpoints in Multiple Myeloma, which brought together clinical investigators in multiple myeloma, the United States Food and Drug Administration, pharmaceutical companies, patient advocates and other concerned scientists and physicians to provide guidance, consensus and consistency in the definition of clinically relevant end points to expedite new drug approvals for multiple myeloma in the appropriate trial design settings. This manuscript will therefore be a most valuable resource to provide the framework for the design of appropriate clinical trial strategies for more rapid new drug approval in myeloma.

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Acknowledgements

We thank James N George, MD, former President of ASH for his inspirational leadership and guidance; and Stephanie Kart at the ASH National Office for her valuable organizational help and support. KCA chaired the Workshop and wrote the manuscript. RAK, SVR, AKS, DW and PR chaired subcommittees and wrote sections of the manuscript.

Author information

Authors and Affiliations

  1. Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA

    K C Anderson & P Richardson

  2. Division of Hematology, Mayo Clinic, Rochester, MN, USA

    R A Kyle & S V Rajkumar

  3. Division of Hematology, Mayo Clinic, Scottsdale, AZ, USA

    A K Stewart

  4. Department of Medicine, MD Anderson Cancer Center, Houston, TX, USA

    D Weber

Authors
  1. K C Anderson
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  2. R A Kyle
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  3. S V Rajkumar
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  4. A K Stewart
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  5. D Weber
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  6. P Richardson
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Consortia

on behalf of the ASHTM /FDA Panel on Clinical Endpoints in Multiple Myeloma

Corresponding author

Correspondence to K C Anderson.

Additional information

This article was adapted from the ASH™/FDA Workshop on clinical end points in multiple myeloma (MM), held in Washington, DC, USA, on October 26, 2006.

Appendix

Appendix

ASH/FDA Panel on Clinical Endpoints in Multiple Myeloma

Workshop Co-Chairs

Kenneth C Anderson, MD, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.

Richard Pazdur, MD, US Food and Drug Administration, Silver Spring, Maryland, USA.

American Society of Hematology Liason

James N George, MD, University of Oklahoma Health Science Center, Oklahoma City, Oklahoma, USA.

Monoclonal Gammopathy of Undetermined Significance/Smoldering Multiple Myeloma Subcommittee.

Chair: Robert A Kyle, MD, Mayo Clinic, Rochester, Minnesota, USA.

Geraldine P Schecter, MD, Veterans Affairs Medical Center Washington, Washington, DC, USA.

Ann T Farrell, MD, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.

Rafael Fonseca, MD, Mayo Clinic, Scottsdale, Arizona, USA.

Newly Diagnosed Multiple Myeloma Subcommittee

Chair: S Vincent Rajkumar, MD, Mayo Clinic, Rochester, Minnesota, USA.

Bart Barlogie, MD, PhD, University of Arkansas for Medical Science, Little Rock, Arkansas, USA.

Sundar Jagannath, St Vincents Hospital, New York, NY, USA.

Kaushikkumar Shastri, MD, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.

Jesus F San Miguel, MD, PhD University of Salamanca, Salamanca, Spain.

Mario Boccadoro, University of Torino, Torino, Italy.

Maintenance Subcommittee

Chair: Keith Stewart, MD, Mayo Clinic, Scottsdale, Arizona, USA.

Peter F Bross, MD, Center for Biologics Evaluation and Research, US Food and Drug Administration, Rockville, Maryland, USA.

Nikhil C Munshi, MD, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.

Jean-Luc Harousseau, University of Nantes, Nantes, France.

Relapsed Multiple Myeloma Subcommittee

Chair: Donna Weber, MD, MD Anderson Cancer Center, Houston, Texas, USA.

Brian Durie, MD, Cedars Sinai Medical Center, Los Angeles, California, USA.

Vicki L Goodman, MD, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.

Sagar Lonial, MD, Emory University, Atlanta, Georgia, USA.

Robert Z Orlowski, MD, PhD, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.

Melissa Alsina, H Lee Moffitt Cancer Center, Tampa, Florida, USA.

Relapsed Refractory Multiple Myeloma Subcommittee

Chair: Paul G Richardson, MD, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.

Joan Blade, MD, Hospital Clinic of Barcelona, Barcelona, Spain.

Donna Reese, MD, Princess Margaret Hospital, Toronto, Ontario, Canada.

William Dalton, MD, H Lee Moffitt Cancer Center, Tampa, Florida, USA.

Seema Singhal, MD, Lurie Cancer Center, Northwestern University, Chicago, Illinois, USA.

Edvardas Kaminskas, MD, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.

Mohamad Hussein, MD, Cleveland Clinic, Cleveland, Ohio, USA.

US Food and Drug Administration Representative

Robert Kane, MD, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.

Industry Representative

Robert J De Lapp, MD, PhD, Celgene Corporation, Summit, New Jersey, USA.

National Institutes of Health Representative

Donna Griebel, MD, Division of Cancer Prevention, National Cancer Institute, Rockville, Maryland, USA.

Patient Representatives

Bruce Holmberg, Rockville, Maryland, USA.

James L Omel, MD, Grand Island, Nebraska, USA.

Statisticians:

John D Crowley, PhD, Southwest Oncology Group Statistical Center, Seattle, Washington, USA.

Rajeshwari Sridhara, PhD, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.

Representatives from Advocacy Groups

Anne Quinn Young, MPH, Director of Programming, Multiple Myeloma Research Foundation.

Brian Durie, MD, Chair, Board of Directors, International Myeloma Foundation.

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Anderson, K., Kyle, R., Rajkumar, S. et al. Clinically relevant end points and new drug approvals for myeloma. Leukemia 22, 231–239 (2008). https://doi.org/10.1038/sj.leu.2405016

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